Setting a New Trend in Rare Disease R&D: Turning Small Data into Long Data

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The Vitiligo Research Foundation's bio-IT system is using Real World Data to give a real boost to researchers in the fight against rare and neglected diseases.

The Vitiligo Research Foundation (VRF) is seeing the massive opportunity in collecting cheap Longitudinal Data as opposed to costly Big Data in order to expedite R&D in rare diseases, using standard healthcare and bioinformatics tools.

Conventional wisdom dictates that data analytics can make typically $1.4 billion drug development faster and cheaper. Big Data deploys sophisticated analytics to parse huge quatities of data from many disparate sources across the healthcare ecosystem to discover patterns that could be useful in problem solving.

The major problem with the current Big Data mega-trend, one that is dominating media casts and conferences nowadays, is that there is not enough data to crunch in a field of more than 7,000 rare diseases. An only slightly lesser problem is that, even if there was enough Big Data on rare diseases, it would be made up of millions of loosely related disease data snapshots, like a movie reel where each frame is technically correct but has no real relation to the surrounding frames.

While Big Data trend may lead to exciting payoffs down the road, the VRF decided not waiting years for hospital-based Electronic Health Record (EHR) platforms to collect meaningful data on this neglected and under-reported disease, and has started with leveraging smaller data.

VRF now collects longitudinal patient records in order to chart the course of a slowly progressing disease more precisely, through using participating clinics, a federated network of biobanks, and online tools like self-reported patient records that feed into the bioinformatics platform, dubbed CloudBank. The upcoming release 2.0 addresses some of the issues reported by testers of the beta-release and expands its functionality. It was outsourced to Carbon Valley, a dynamic Russian IT company focused on Real World Data collection and analytics for bio-pharma and healthcare industries.

The VR Foundation’s CEO Yan Valle has an engineering and traditional computer science background and, in fact, started working for the Foundation thinking that they were going to be focused on bioinformatics. Ultimately, the VRF’s team has built a disease-specific EHR system linked to a dozen biobanks across the world, a trend that wasn’t in vogue at the time of its inception, but is being more and more recognized as an essential part of disease research. "He is an inspiration for us to pursue this trend," says the Dmitry Aksenov, VRF Founder and President. "With biopharmaceutical industry becoming increasingly risk‐averse, the timing is right for new models on how to do research," he adds.

"There are many foundations funding research on different diseases, but fewer that are investing in the infrastructure to expedite and manage that research process more effectively," says Dr. Igor Korobko, VRF Chief Scientific Director. "We funded the Vitiligo Biobank network in part because it will provide us with desperately needed research-quality data. Thus, it will produce quality results in both understanding the disease and faster development of new treatments for the millions of people suffering from disease."

About Vitiligo: Vitiligo is an under-investigated skin disease that affects between 65 and 95 million people worldwide. Vitiligo is a generally unpredictable disease, which results in a loss of inherited skin color over the lifetime. It is non-lethal and it does not cause any organic harm, but it has a devastating socio-economic effect on the affected person and their family, especially in darker skinned populations.

About the Vitiligo Research Foundation. The VR Foundation is a 501(c)3 non-profit, disease-specific foundation. As the nation's only organization addressing every phase of vitiligo treatment development, VRF works with a diverse group of support organizations and individuals to achieve measurable and timely results. When it comes to finding a cure for vitiligo, the VRF expects to make a difference in our lifetime.


Yan Valle
CEO, Vitiligo Research Foundation
1, Penn Plaza #6205, New York, NY

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